,33,34,35 Remarkably, the long-lasting sclerostin aptamer for osteogenesis imperfecta (OI) was granted Rare Pediatric Disease Designation (RPD-2022-667) and Orphan Drug Designation (DRU-2022-9087) by the US Food and Drug Administration (FDA) in 2022. The gene discussed is SOST; the disease is osteogenesis imperfecta.